Entries in Drug Shortages
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St. Jude's Children's Research Hospital(NEW YORK) -- Abby Alonzo was eight weeks into treatment for Hodgkin lymphoma and doing great when her doctor informed the 9-year-old's parents that one ingredient in the cocktail of chemo drugs she was receiving was no longer available and they would have to use a substitution.

"It's scary, and I was alarmed but I didn't really have a sense of how critical this drug was," said Katie Alonzo, Abby's mother. "When something like this happens, you have to put your faith in the doctors."

But by 12 weeks into treatment, Abby relapsed. Rather than receiving a planned break from treatment, she was rushed into emergency "salvage therapy," which involved more chemo, a bone marrow transplant and radiation.

The doctors at St. Jude Children's Research Hospital in Memphis, Tenn., who treated Abby, attribute her relapse to the shortage of the drug mechlorethamine, also known as nitrogen mustard.

In Thursday's New England Journal of Medicine, the St. Jude doctors, along with colleagues from the Dana-Farber cancer institute in Boston and the Lucile Packard Children's Hospital in Stanford, Calif., highlight how the scarcity of this medication has been linked to a higher rate of relapse among children, teenagers and young adults with Hodgkin lymphoma.

The 40 patients in the study who received the substitute drug cyclophosphamide experienced complications at about twice the rate of the 181 patients studied who didn't have to switch drugs. This is the first randomized study to compare the differences in treatment outcomes between the two medications.

None of the patients died, but the authors emphasize that they're only 18 months out from treatment and could still be hit with adverse effects.

"It's too soon to know about problems and success rates," said Dr. Michael Link, one of the lead investigators and a Stanford University pediatric oncologist. "Even if they're cured, they underwent intensive treatment they would have otherwise avoided and this puts them at greater risk for long-term effects such as infertility and secondary cancers."

Dr. Amy Billet, a pediatric oncologist at Dana-Farber who was also part of the study, said it's not possible to say beyond a shadow of a doubt that the drug swap was responsible for the higher rate of relapse, but she and her fellow investigators could think of no other credible explanation.

"You can never know what happened to one individual patient. You can only say there is an increase risk in the group who got the substitution drug," she said.

According to Link, this latest study only confirms what doctors have suspected for years: swapping one drug for another, even when the two are similar, isn't always ideal.

"We thought this one was a no-brainer because the drugs were so alike, but we discovered to our dismay that the substitution didn't work as well. This takes the problem out of the hypothetical and we can point our finger at exactly which patients suffered as a result of the shortage," he said.

Reasons for the shortage aren't entirely clear. It's likely a combination of factors such as limited manufacturing, lagging production time and lack of profits from these drugs.

The Food and Drug Administration has tried to step in to remedy the problem. For example, after President Obama issued an executive order in 2011 to reduce the dire drug shortages, the FDA broadened its reporting of potential drug shortages and expedited regulatory reviews that sometimes made the shortages worse.

The New England Journal of Medicine authors said the FDA's efforts should be commended but said more needed to be done. They said that the shortage of mechlorethamine eased up shortly after their study ended, with no explanation. However, they warn there's no guarantee there won't be another shortage later.

Pixland/Thinkstock(PARK RIDGE, Ill.) -- Seven U.S. anesthesiologists have reported that drug shortages resulted in deaths of their patients, according to a new survey from the American Society of Anesthesiologists.

Although the online survey completed by 3,063 of the organization's 28,000 practicing anesthesiologists cannot be considered scientific, it opens a window to the growing shortage of anesthetics and painkillers used before, during and after surgery, and the potentially fatal consequences when those are unavailable.

Seven doctors responded to the question, "How has a drug shortage impacted your patients?" by checking the option, "Has resulted in death of a patient," according to survey results exclusively obtained by ABC News.

"We have a warning here. We are bound to trace this warning down and find out the extent of it," American Society of Anesthesiologists President Dr. Jerry A. Cohen said in an interview. "Are these the canary in the mine? I don't know. I worry that they are."

Because the survey was anonymous, Cohen said he couldn't determine where the reported deaths occurred, how the patients died, or be sure all seven reports were reliable. "Six we're absolutely sure were reported," Cohen said.

ASA had questions about "data points" in one responder's answers that cast doubts on one of the death reports, he explained. "It's unusual to have anybody report a death due to a drug shortage," Cohen said.

Responses to a similar ASA survey in 2011 included two reports of patient deaths, but those weren't made public, Katherine Looze, an ASA spokeswoman, revealed in response to a question from the ABC News Medical Unit.

Going forward with surgeries when they're short of needed drugs can put anesthesiologists in a legal bind, Cohen said.

"We are at risk," he added. "If I proceed, if I don't have the drug I need -- and know I don't have the drug I need -- and I have a bad outcome, I'm responsible."

Among survey respondents -- 3,033 Americans and 30 from abroad -- 97.6 percent reported being currently short of at least one drug and 96 percent said shortages forced them to use substitutes for particular patients and procedures. When they used alternatives to their top-choice drugs, some of their patients experienced nausea and vomiting, spent longer in surgery or took longer to recover from sedation or anesthesia, the survey revealed.

The drugs survey respondents most often couldn't get were the workhorses of local and general anesthesia, such as propofol, a milky white intravenous sedative and anesthetic that works in just 40 seconds. It's best known as the drug that led to the death of pop superstar Michael Jackson. Another is succinylcholine, a quick-acting paralytic agent frequently given to help insert a breathing tube into a patient's airway. There's also fentanyl, a powerful synthetic opioid used to knock patients out and to treat operative and post-op pain.

Anesthesiologists also reported problems getting thiopental, also known as sodium penthothal, an ultra-short acting barbiturate that's used less frequently today in hospitals, but remains in demand for the lethal cocktail many states use in executions. The shortage has left some prisoners longer on death row.﻿

iStockphoto/Thinkstock(BALTIMORE) -- University of Maryland head women's basketball coach Brenda Frese is not afraid of challenging authority, especially off the court when it involves her 4-year-old son, Tyler, who has leukemia. Frese was shocked when she discovered that the treatment he required was threatened by a cancer drug shortage.

“Let’s wake up here,” Frese told ABC News. “How many people have to die....It makes me sick to my stomach to think about it.”

So Frese wrote to her congressman, Elijah Cummings, D-Md., and found out that fake pharmacies are one big reason for the shortage.

“What they do is, they horde the drugs and basically create the shortage,” Cummings said. “Not only is it insensitive, it’s criminal.”

Here’s how it works: The fake pharmacy, which is licensed, acts as a middleman. But rather than dispensing short-supply drugs to patients, as their licenses require, they buy drugs from the manufacturer, then sell the drugs to a wholesale company they also own. The wholesale company, which is often at the same address as the fake pharmacy, then offers the drugs to desperate hospitals, often at an even higher price.

Investigators say fake pharmacies sold a $15 vial of cancer-fighting fluorouracil to hospitals for $350.

Congress sent 19 letters to supposed pharmacies across the country this week and are working with state authorities to pull their licenses. North Carolina closed one that never actually saw any customers, and whose shelves had few drugs, only those that were in short supply.

“We’re paying a ridiculous price for many of these drugs, sometimes marked up 100, 200, 300 percent for a drug that we get for a couple of bucks,” said Dr. Jennifer Brandt, clinical specialist pharmacist at Washington Hospital Center.

iStockphoto/Thinkstock(WASHINGTON) -- The U.S. Food and Drug Administration approved new suppliers for two cancer drugs Tuesday in an effort to curb the largest nationwide drug shortage in nearly a decade.

The FDA said its approval of a new supply method would increase production of the injection form of the drug methotrexate, which is used to treat children with the most common form of childhood leukemia.

The agency also said it would allow temporary foreign imports of the cancer drug Lipodux, an alternative to the drug doxorubicin, sold under the name Doxil, which is used to treat ovarian cancer, multiple myeloma and AIDS-related Kaposi's sarcoma. Temporary foreign importation is a rare move for the agency.

"In different circumstances we have to apply different tools," said FDA Commissioner Peggy Hamburg in a statement, adding that the quality of the drug was evaluated by the agency before its temporary approval.

The FDA's actions follow President Obama's executive order issued in October 2011 to reduce the dire drug shortage. The order instructed the FDA to broaden its reporting of potential drug shortages, expedite regulatory reviews that can help prevent shortages and investigate whether potential shortages have led to price gouging.

The drug shortage has compromised or delayed care for some patients and may have led to otherwise preventable deaths, say many oncologists.

All five pharmaceutical companies that make the injection drug methotrexate, which treats acute lymphoblastic leukemia by slowing the growth of cancer cells, have either slowed or stopped manufacturing of the drug, according to the FDA. The companies have blamed shortages on high demand or manufacturing delays.

Drug manufacturers APP Pharmaceuticals and Hospira Inc. announced at the press briefing Tuesday that within four to six weeks, both companies would have made enough methotrexate, including the preservative-free form, to meet the nationwide demand.

Next week Hospira plans to release 34,000 vials of methotrexate, or a month's supply of the drug, Michael Ball, CEO of Hospira Inc., told reporters at Tuesday's briefing.

According to Michael Link, a pediatric oncologist and president of the American Society of Clinical Oncology, some hospital pharmacies reported having only a couple of weeks of supplies left.

Many oncologists are especially worried about the shortage of the preservative-free form of methotrexate, which is considered less toxic.

Only the preservative-free methotrexate can be injected into the spinal fluid of cancer patients to prevent the spread and recurrence of the disease.

"There are couple other drugs that can be injected into the spinal fluid, but none that are as effective," said Link. "As for the high dose version of the drug, there's no workaround for it."

Sen. Orrin Hatch, R-Utah, and Sen. Charles Grassley, R-Iowa, both expressed criticism and concern about the contraceptive mandate. Hatch questioned whether Sebelius had consulted with any Catholic bishops on the matter prior to last week’s reversal on the mandate that would require religious employers to cover certain preventable health services, such as contraception.

“I did not,” she said, adding that the president has, “spoken to the bishops on several occasions,” but she was unsure if it was about the compromise in the requirement.

The comprimise touted by the administration after a firestorm erupted over the mandate did little to quell the controversy. This week, the nation’s Roman Catholic bishops rejected the Obama administration's compromise on birth control coverage and said they would continue to fight President Obama's plan to force employees of Catholic hospitals, universities and other institutions to provide free contraceptive coverage in their health insurance plans for their employees.

Hatch also pressed her on whether HHS had conducted or requested any, “analysis of the constitutional or statutory religious freedom issues” surrounding the mandate. Sebelius replied that she never spoke to anyone, but that HHS did, “look at a whole host of legal issues.”

Pressing the issue, Hatch asked if HHS consulted anyone at the Justice Department, to which Sebelius said “no.”

“I think you’ve got it very wrong the first try,” Grassley said, before turning his attention to whistle-blower protection. “You have a lot more work to do.”

But it wasn’t all negative surrounding the mandate. Sen. Jeff Bingaman, D-N.M., commended the “reasonable position” HHS has taken.

“I think that it adequately protects religious liberty and it at the same time protects the right of women to obtain contraceptive services when they choose to,” Bingaman said.

The recent drug shortages making headlines across the nation were also a topic of discussion.

Citing a 3-year-old in his district who can’t get her leukemia medication, Sen. Ron Wyden, D-Ore., questioned why the government can’t, “fix this and stop playing catch-up ball.”

“The FDA did announce yesterday that they feel that in the next two weeks the leukemia drug shortage will indeed be resolved,” Sebelius said. “It’s resolved because what we can do at the FDA is accelerate alternatives, if we have notification.”

Sebelius cited a “market glitch” as the main cause of the problem.

“The market capacity for drugs has not increased,” she said. “Currently, we have the same manufacturing capacity and drug marketers choosing which line of drugs to produce at which time.”

A bill currently pending in the House and Senate would make it a requirement for drug companies to notify the government of an impending shortage -- a process Sebelius said was “key” for the government’s role in preventing shortages.﻿

iStockphoto/Thinkstock(WASHINGTON) -- The U.S. Food and Drug Administration said Tuesday that a shortage on the life-saving drug used in childhood leukemia treatments could be averted.

Last week, doctors spoke out about the looming shortage of methotrexate -- a key drug known to help cure some young patients with pymphoblastic leukemia when combined with other drugs in high doses. Some oncologists expressed worry the drug may run out in as little as two weeks, HealthDay reports.

But Tuesday, the FDA showed more optimism.

"We are seeing the companies [that make methotrexate] respond to this shortage and they are planning on some very large releases, and we are planning on having the situation resolved," Valerie Jensen, associate director of the FDA's drug shortage program, said Tuesday, according to HealthDay.

Hospira, one of the three manufacturers of the drug based in Illinois, has said the company will boost production of the drug to "make up for the gap," HealthDay reported Tuesday. The other two makers of the crucial drug -- Mylan Inc. in Pennsylvania and Sandoz US Inc. in New Jersey -- both say they also plan to increase methotrexate production.

iStockphoto/Thinkstock(HOUSTON) -- Medication used to treat the most common form of childhood leukemia is in short supply, adding to the largest nationwide shortage of critical lifesaving hospital medications in nearly a decade.

All five pharmaceutical companies that make the injection drug methotrexate, which treats acute lymphoblastic leukemia by slowing the growth of cancer cells, have either slowed or stopped manufacturing of the drug, according to the U.S. Food and Drug Administration. The companies have cited high demand or manufacturing delays as reasons for the shortage.

If the shortage continues, physicians and pharmacists fear thousands of children will be left without lifesaving treatment.

"This, to us in oncology, is a national crisis," said Brooke Bernhardt, clinical pharmacy specialist in the department of hematology and oncology at Texas Children's Hospital in Houston.

According to Dr. Michael Link, pediatric oncologist and president of the American Society of Clinical Oncology, some hospital pharmacies have reported having only a couple weeks of supply left.

Many oncologists are especially worried about the shortage of the preservative-free form of methotrexate, which is considered less toxic.

Only the preservative-free methotrexate can be injected into the spinal fluid of cancer patients to prevent the spread and recurrence of the disease.

Each year, nearly 3,000 children and adolescents under age 20 are diagnosed with acute lymphoblastic leukemia in the United States, according to the National Cancer Institute. Eighty percent of children are successfully treated.

The agency said it's uncertain when the next batch of methotrexate will be available.

Drugs for heart patients, some antibiotics, and intravenous drugs have been hard for hospitals to find, but cancer drugs have arguably taken the hardest hit.

To compensate, some physicians have had to split vials among patients or use comparable medications. In some cases, physicians may triage the medication, delaying treatment for some who may not need it immediately.

President Obama issued an executive order in October 2011 to reduce the dire shortage. The order instructed the Food and Drug Administration to broaden reporting of potential drug shortages, expedite regulatory reviews that can help prevent shortages, and examine whether potential shortages have led to price gouging.

The drug shortage has compromised or delayed care for some patients and may have led to otherwise preventable deaths.

While the FDA can oversee imports of drugs that are in short supply, it cannot regulate how much a company can make. In fact, manufacturers are not required to report shortages to the FDA.

The amount of a drug made available within a hospital is set by an agreement between the hospital and the manufacturer.

Limited manufacturing, lagging production time and lack of profits from these drugs contribute to the shortages. The production costs for some drugs can outweigh the money that companies can make from them, since many drugs now have cheaper generic alternatives. So manufacturers stop making the drugs.

Since these medications are mainly housed in hospitals, most patients won't know they can't have them until they really need them.

"Many physicians may be willing to mention the shortage to the family because they're just as frustrated," said Bernhardt.

However, Bernhardt said, that supply counts depend on location. Some hospitals may not be experiencing a shortage at all, she said.

"We don't want to stir up fear, but [families] should remain in contact with their physician," said Bernhardt.

iStockphoto/Thinkstock(NEW YORK) -- A contentious relationship between drug manufacturers and the Drug Enforcement Agency may cause a continuing shortage of the attention deficit medication Adderall, which the Food and Drug Administration just added to its official drug shortages list, the New York Times reported.

As of 2007, about 9.5 percent of school-aged children, or 5.4 million, were diagnosed with Attention Deficit Hyper Disorder (ADHD), according to the Centers for Disease Control and Prevention. Since then, prescriptions for Adderall have numbered in the millions and continue to increase. And as demand for the drug grows, more and more patients have found the medication is out of stock at local pharmacies.

Experts say it’s difficult to say where the reason for drug shortage lies. To manage controlled substances that can potentially be abused, the DEA sets manufacturing quotas for drug ingredients each year to control supplies like Adderall. But Adderall drug manufacturers, which include Shire Plc and Novartis, Teva and CorePharma LLC, say they cannot meet the growing demand for the product without looser limits from the DEA.

The DEA questions whether there is actually a shortage of generic supplies, which are at an especially low supply, or whether the drug companies want to sell more of the expensive brand-name drugs.

Despite the growing demand, Special Agent Gary Boggs of the DEA’s office of diversion control told the New York Times, "We believe there is plenty of supply."

Comstock/Thinkstock(WASHINGTON) -- A growing shortage of important chemotherapy drugs, anesthetics and antibiotics, which has compromised or delayed care for some U.S. patients and may have led to at least 15 deaths, represents a "pressing public health problem," a top federal health official said Friday.

At the same time, shortages of medications to treat cancer and infectious diseases have strained, disrupted or derailed hundreds of important research trials within the National Institutes of Health, said Dr. Howard K. Koh, assistant secretary of the Department of Health and Human Services. He called the disruptions "very troubling to us as a nation that prides itself on scientific advances."

Koh painted a picture for the House Energy and Commerce Subcommittee on Health in which even the Food and Drug Administration, the federal agency responsible for assuring the safety of the U.S. drug supply, has been caught unaware when particular drugs have become scarce -- or impossible to get. "Oftentimes, the FDA does not know until it is too late, and then patients are stuck in this dire situation, and that's just not acceptable," Koh told lawmakers.

"We still have a large percentage of actual shortages where we were not aware that it was coming," Dr. Sandra Kweder, deputy director of the Office of New Drugs in the FDA's Center for Drug Evaluation and Research, told the panel.

One possible solution to the problem would be to find a way for manufacturers "to report impending supply disruptions and discontinuation of drugs," Koh said. "The sooner FDA learns of a drug shortage, the more effective it can be in helping to notify providers and minimizing the impact on patients."

The FDA doesn't have the power to require companies to report impending shortages. However, the proposed Preserving Access to Life-Saving Medications Act would give the agency the authority to require advance notification from drug manufacturers when life-saving drugs are in danger of becoming unavailable. The American Cancer Society Cancer Action Network, an affiliate of the American Cancer Society, Friday announced its support for the legislation.

As of Friday, 209 drugs were on the current shortage list compiled by the American Society of Health-System Pharmacists, based in Bethesda, Md., and some of them are the heaviest-hitters in cancer treatment and the battles against increasingly drug-resistant antibiotics.

Paul Tearle/Stockbyte/Thinkstock(PITTSBURGH) -- Jerry Walter, a semi-retired Army colonel who lives in North Carolina, said he has led a "charmed life," surviving the terrorist attack on the Pentagon in 2001 and two life-threatening bacterial infections.

At 56, Walter has also outlived a brother and two other relatives who, like him, had Fabry disease, a rare genetic disorder that can wreak havoc on the kidneys and cause strokes and early death. Caused by the lack of an enzyme -- alpha-galactosidase-A -- which is required to metabolize lipids or fatlike substances in the body, its symptoms include chronic gastrointestinal and cardiovascular problems, pains in the hands and feet, renal failure, as well as skin and eye complications, according to the National Institutes of Neurological Disorders and Stroke.

Since 2003, the expensive drug Fabrazyme has kept Walter alive, but since 2009, Fabrazyme has been in short supply, and his dosage has been cut in half.

"I began to have a decline in kidney function and more arrhythmia after the shortages," said Walter, who is founder of the Fabry Disease Foundation.

Jeanette Hyatt, 33, of Royal Oak, Michigan, is currently recovering from a stroke after going two years without full dosages of Fabrazyme.

"I am hanging in there, but I have trouble with my speech," said Hyatt, who is on disability. "It's crazy. I talked to the FDA, but they can't force a drug manufacturer to do anything."

Since early August, Genzyme, the manufacturer of Fabrazyme, has not been able to ship the drug to an estimated 1,200 Americans with the disease, and now patients fear that shipments will be stopped altogether in September.

Now, Hyatt is one of 14 patients who are suing Genzyme for physical damages because of the drug's unavailability. They have also petitioned the Food and Drug Administration to impose a ban on the sale of the drug outside the United States -- Europeans are receiving full dosages of Fabrazyme as Americans get sicker.

Their lawyer, C. Allen Black of Pittsburgh, said many more will soon be joining the lawsuit.

"This is one of the worst avoidable tragedies in American medicine, and the suffering has been incalculable," according to Black, who said patients are suing for millions of dollars in damages because they are so sick they cannot work.

The drugs cost $30,000 for each infusion, and a full dose consists of two infusions a month.